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Substrate Reduction Therapy

Eliglustat +/− Imiglucerase for Gaucher Disease (ELIKIDS Trial)

Q: Has Imiglucerase GZ437843 received authorization from the FDA?

Imiglucerase GZ437843 is a Phase 3 trial medication, meaning that its efficacy has been supported by some data and its safety has been corroborated through multiple rounds of testing.

Q: Are there any other notable clinical trials that have used Imiglucerase GZ437843?

Right now, 4 different research groups are studying Imiglucerase GZ437843. 1 of those trials is in the advanced Phase 3 stage. Even though most of the clinical trials for Imiglucerase GZ437843 take place in Toronto and Saitama, there are a total 60 research sites running these tests.

Q: What benefits does this research hope to bring about?

The researchers conducting this study will primarily be assessing the level of a particular protein over the course of the trial. Additionally, they will also be looking for improvements in bone disease, quality of life, and changes in hemoglobin levels.

Q: What is the primary disease that Imiglucerase GZ437843 has been shown to improve?

Imiglucerase GZ437843 is used to treat cyp2d6 poor metaboliser status and can also ameliorate the symptoms of gaucher disease, type 1, gaucher disease, type 3, and no neurological problems.

Q: Could you explain the patient criteria for this research project?

This study is seeking 57 paediatric patients, aged 2-17, who have been diagnosed with a specific disease. In order to be eligible for the trial, participants must also meet the following criteria: postmenarchal female patients must have a negative <a href="https://www.withpower.com/clinical-trials/pregnancy">pregnancy</a> test on record prior to enrolling in the study and throughout its duration; patients must be willing to practice true abstinence or use an accepted form of contraception throughout the study; liver volume must be less than 1.5 MN; absence of GD related pulmonary disease; and severe bone disease, as defined below for Cohort 2. Additionally, spleen volume must be less than 10

Phase 3

Waitlist Available

Research Sponsored by Sanofi

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

The patient is 2 to <18 years old at the time of informed consent.

Male and female patients with a clinical diagnosis of Gaucher disease (GD) type 1 or type 3 with documented deficiency of acid beta-glucosidase activity by enzyme assay and glucocerebrosidase (GBA) genotype.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up weeks 2, 13, 26 and 52

Awards & highlights

ELIKIDS Trial Summary

This trial will test the safety and effectiveness of a drug called eliglustat in children aged 2 to 18. Researchers will also look at the drug's effects on quality of life.

Who is the study for?

This trial is for kids aged 2-17 with Gaucher Disease types 1 or 3. They must have been on enzyme replacement therapy (ERT) and meet certain health criteria. Girls post-puberty need a negative pregnancy test and agree to contraception or abstinence.Check my eligibility

What is being tested?

The study tests the safety and effects of Eliglustat alone, or combined with Imiglucerase in children with Gaucher Disease. It looks at how their bodies handle the drugs (pharmacokinetics), changes in their condition, and life quality.See study design

What are the potential side effects?

Possible side effects include allergic reactions, especially if there's a known sensitivity to Imiglucerase. Other risks may involve digestive issues, fatigue, headaches, but specific side effects will be monitored throughout the study.

ELIKIDS Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I am between 2 and 17 years old.

Select...

I have been diagnosed with Gaucher disease type 1 or 3.

ELIKIDS Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ weeks 2, 13, 26 and 52

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~weeks 2, 13, 26 and 52

This trial's timeline: 3 weeks for screening, Varies for treatment, and weeks 2, 13, 26 and 52 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Adverse Events

Assessment of PK parameter of eliglustat: AUC

Assessment of pharmacokinetic (PK) parameter of eliglustat: Cmax

Secondary outcome measures

Bone disease improvement

Change in hemoglobin level

Change in liver volume

+5 more

ELIKIDS Trial Design

2Treatment groups

Experimental Treatment

Group I: Cohort 2: Eliglustat plus imigluceraseExperimental Treatment2 Interventions

Eliglustat plus imiglucerase for three years, at the dose of enzyme replacement therapy received before enrollment. After Week 52, Cohort 2 patients will switch to eliglustat monotherapy for the remainder of the study if the desired clinical response has been achieved.

Group II: Cohort 1: Eliglustat monotherapyExperimental Treatment1 Intervention

Eliglustat for at least two years. Cohort 1 patients that experience significant clinical decline will receive rescue treatment. Rescue Treatment Step 1: Switch from eliglustat to imiglucerase monotherapy. Rescue Treatment Step 2: Patients who after 6 months of rescue therapy with imiglucerase monotherapy do not show improvement in the parameter(s) that led to the switch from eliglustat to imiglucerase, will then receive combination therapy with eliglustat + imiglucerase.

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

SanofiLead Sponsor

2,164 Previous Clinical Trials

3,514,923 Total Patients Enrolled

13 Trials studying Gaucher Disease

6,933 Patients Enrolled for Gaucher Disease

Clinical Sciences & OperationsStudy DirectorSanofi

862 Previous Clinical Trials

2,019,781 Total Patients Enrolled

10 Trials studying Gaucher Disease

920 Patients Enrolled for Gaucher Disease

Media Library

Eliglustat GZ385660 (Substrate Reduction Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03485677 — Phase 3

Gaucher Disease Research Study Groups: Cohort 1: Eliglustat monotherapy, Cohort 2: Eliglustat plus imiglucerase

Gaucher Disease Clinical Trial 2023: Eliglustat GZ385660 Highlights & Side Effects. Trial Name: NCT03485677 — Phase 3

Eliglustat GZ385660 (Substrate Reduction Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03485677 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has Imiglucerase GZ437843 received authorization from the FDA?

"Imiglucerase GZ437843 is a Phase 3 trial medication, meaning that its efficacy has been supported by some data and its safety has been corroborated through multiple rounds of testing."

Answered by AI

Are there any other notable clinical trials that have used Imiglucerase GZ437843?

"Right now, 4 different research groups are studying Imiglucerase GZ437843. 1 of those trials is in the advanced Phase 3 stage. Even though most of the clinical trials for Imiglucerase GZ437843 take place in Toronto and Saitama, there are a total 60 research sites running these tests."

Answered by AI

What benefits does this research hope to bring about?

"The researchers conducting this study will primarily be assessing the level of a particular protein over the course of the trial. Additionally, they will also be looking for improvements in bone disease, quality of life, and changes in hemoglobin levels."

Answered by AI

What is the primary disease that Imiglucerase GZ437843 has been shown to improve?

"Imiglucerase GZ437843 is used to treat cyp2d6 poor metaboliser status and can also ameliorate the symptoms of gaucher disease, type 1, gaucher disease, type 3, and no neurological problems."

Answered by AI

Could you explain the patient criteria for this research project?

"This study is seeking 57 paediatric patients, aged 2-17, who have been diagnosed with a specific disease. In order to be eligible for the trial, participants must also meet the following criteria: postmenarchal female patients must have a negative pregnancy test on record prior to enrolling in the study and throughout its duration; patients must be willing to practice true abstinence or use an accepted form of contraception throughout the study; liver volume must be less than 1.5 MN; absence of GD related pulmonary disease; and severe bone disease, as defined below for Cohort 2. Additionally, spleen volume must be less than 10"

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3

2018. "Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03485677.

All > Gaucher Disease